Minister Ambrose Announces Patient Involvement Pilot for Orphan Drugs

Input will help shape the role of public contributions to orphan drug
approvals

OTTAWA, Aug. 6, 2014 /CNW/ - The Honourable Rona Ambrose, Minister of
Health, announced today that the Government of Canada has launched a
pilot project targeting patient input from Canadians with rare diseases
to help inform future reviews of orphan drugs. As announced in October
of 2012, Health Canada has been developing an Orphan Drug Framework to
spur innovation and research into new treatments for rare diseases and
also to encourage patient participation. The Pilot Project will
simulate how input from patients will be gathered and incorporated into
the drug submission review process once the Orphan Drug Framework is in
effect.

Drug manufacturers, Hoffmann-La Roche Limited and Hyperion Therapeutics,
Inc have agreed to participate in the pilot with two of their drug
review submissions. The review of Hoffmann-La Roche's medication
obinutuzumab for the treatment of chronic lymphocytic leukemia (CLL)
will be used for the pilot. Hyperion Therapeutics Inc.'s review is for
Ravicti ® (glycerol phenylbutyrate) Oral Liquid which is used for the
treatment of urea cycle disorders. Hoffmann-La Roche and Hyperion's
participation will serve as models for how patient input may inform
drug authorization decisions.

Patients will be asked to comment on the following:

how the rare disease affects their ability to manage their day-to-day
lives;

what treatments are currently available (if any);

what therapeutic benefits are most important to them; and

their risk tolerance for any new treatments.

Health Canada will be asking patient advocacy groups including the Canadian Organization for Rare Disorders to assist in engaging specific Canadian patients affected by CLL or
urea cycle disorders to participate in the project. The patient
feedback provided during the pilot will assist Health Canada in
assessing and refining its approach to gathering patient input. This
will ensure that patient perspectives are considered in future orphan
drug authorizations.

Quick Facts

Orphan drugs are those drugs used to treat rare diseases. A rare disease
is a life-threatening, seriously debilitating, or serious chronic
condition that only affects a very small number of patients (typically
less than 5 in 10,000 persons).

When the Orphan Drug Framework is fully implemented, Canadian patients
will be invited to participate in future orphan drug authorizations
through Health Canada's website.

Patients with CLL or urea cycle disorders, their current or former
caregivers or healthcare professionals, and others with first-hand
knowledge of CLL or urea cycle disorders are encouraged to contact
Health Canada by email if they wish to participate at: odpp.ppmo@hc-sc.gc.ca.

Quotes

"No one has a better understanding of what it means to have a rare
disease than the patients who are affected by them. The input of these
patient volunteers is absolutely invaluable and our government has
committed to making their experiences and perspectives an important
addition to our scientific approach to drug assessments for rare
diseases."Rona AmbroseMinister of Health

"Roche recognizes that patients and patient organizations are playing an
increasingly important role in drug review and health technology
assessment and bring valuable experience, perspective and insight to
the process. As an organization committed to patients, Roche is pleased
to participate in this Health Canada pilot program that is seeking
patient input within the regulatory review process."Lorenzo BiondiVice President, Medical & Regulatory Affairs, Hoffmann-La Roche Limited

"As part of our full commitment to serving the urea cycle disorder
community, we're looking forward to participating in Health Canada's
Patient Involvement Pilot Project. Orphan diseases have a dramatic
impact on the lives they touch, making this initiative critical to the
process of drug review and approval."Dr. Bruce ScharschmidtSenior Vice president, Chief Medical and Development Officer, Hyperion
Therapeutics, Inc.

"Kudos to Health Canada for recognizing the value of the direct patient
voice in the drug review process, for designing a user-friendly
template that will encourage patient engagement, and for piloting this
in advance of the orphan drug regulations. The Canadian Organization
for Rare Disorders is pleased to have had the opportunity to
collaborate on this important initiative and to promote this in the
patient community."Durhane Wong-RiegerPresident, Canadian Organization for Rare Disorders